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Progress in chordoma research serves as a template for other rare cancers

This article holds up the Foundation’s proactive and hands-on approach to enabling and accelerating chordoma research as a template to be followed for other rare cancers.


Since the Chordoma Foundation started, we have been driven by the belief that success in enabling and accelerating research for chordoma would not only lead to the cure we seek, but also inform new approaches for other rare cancers. We are, therefore, delighted that a case study featuring our research model, and the progress it has catalyzed over the past decade, was recently featured in a review article entitled “Emerging opportunities for target discovery in rare cancers,” published in the prominent scientific journal Cell Chemical Biology.

Written by our partners at the Broad Institute of Harvard and MIT – with whom we are engaged in a multi-year collaboration to uncover new therapeutic targets for chordoma – the article holds up the Foundation’s proactive and hands-on approach to enabling and accelerating chordoma research as a template to be followed for other rare cancers.

In the paper, authors Tanaz Sharifnia, Andrew Hong, Corrie Painter, and Jesse Boehm, detail a convergence of technological advances – from better, cheaper DNA sequencing to the emergence of gene editing technologies such as CRISPR – which are changing the universe of possibilities for rare cancer research and are making it possible to uncover potential therapeutic targets with unprecedented efficiency. The paper goes on to illustrate how the Foundation has brought each of these technologies to bear on chordoma in the past decade, resulting in a growing pipeline of promising treatments entering clinical trials.

“Fortunately, these advances in technology are coming at a time when patient-led inquiry is strengthening, and online patient networks are facilitating greater participation in research. Their convergence has created a shift in momentum around precision medicine in the rare cancer community. The Chordoma Foundation has approached these changes in a thoughtful and well-organized way, and the playbook they’ve built can be applied by others to bring new treatments from theory to reality.”
– Jesse Boehm, Associate Director of the Broad Institute’s Cancer Program

A changing outlook

Historically, rare cancers like chordoma have posed a special set of challenges to research due to their low incidence. These include a scarcity of tumor tissue needed to understand the biology of the disease, lack of cell lines and animal models needed for drug testing and other experimentation, and difficulty finding patients to participate in clinical studies.

Because of these challenges, when the Foundation was formed, chordoma research was at a standstill. Only a handful of researchers were studying chordoma, and no companies were investing. As a result, virtually nothing was known about the biology of the disease, only one clinical trial had ever been completed, and no effective drugs were available.

Over the past decade, our investments have been guided by a belief that if we could make chordoma research faster and more efficient, we could attract scientists and companies to invest their time, talent and resources to develop better treatments for this disease. Moreover, we wagered that by alleviating key research roadblocks we could enable chordoma to benefit from the emergence of ever more powerful technologies that are revolutionizing cancer research.

Today, that wager is paying off.

More than 300 researchers and a dozen companies have joined the search for a cure, and a suite of research-enabling programs has overcome many of the barriers and inefficiencies that once stood in the way of progress.

“Because of the Foundation’s efforts, cutting-edge technologies and approaches are being applied to the study of chordoma. As a result, several potential therapeutic targets have been identified, and a growing number of candidate therapies are being advanced from the laboratory into clinical trials.”
– Tanaz Sharifnia, Research Scientist, Broad Institute

Rare cancer template

Chordoma is one of more than 300 rare cancers, which, together, account for a quarter of all cancer cases, and represent the fourth leading cause of death in developed nations. While research for chordoma and a handful of other rare cancers has bounded forward in recent years, many rare cancers have yet to benefit from similar advances. That may now start to change as the approaches described by our Broad Institute collaborators are applied more broadly.

We have long hoped and believed that lessons gleaned from our experience battling chordoma would inform the broader fight against rare cancers, and this paper is an encouraging sign that that vision is coming to fruition.

“When there are better therapies and a cure for chordoma, it will be directly traceable back to the Foundation’s – and more pointedly, the patients’ – involvement.”
– Corrie Painter, Associate Director of Operations and Scientific Outreach, Broad Institute

Read the full Cell Chemical Biology article »
Learn about our partnership with the Broad Institute »

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